Who Can Participate?
The reLiver-1 trial is looking for babies both with and without a Urea Cycle Disorder. Up to 30 babies will be enrolled, aged birth to 12 months: 20 babies with UCD and 5-10 babies without UCD.
General Participation Requirements
[Specific requirements can be confirmed directly by the hospital or through the website contact form]
Babies with UCD
Your baby can take part in the reLiver-1 trial if:
- They are in the 5th to 95th percentile of their age-related target bodyweight.
- They are medically stable.
- They have one of the 4 gene defects required by the study:
- Deficiency of ASS (Argininosuccinic acid synthetase).
- Deficiency of ASL (Argininosuccinic acid lyase).
- Deficiency of CPS1 (Carbamoylphosphate synthetase I).
- Deficiency of OTC (Ornithine transcarbamylase).
If the gene defect test result is not available from your baby’s regular doctor, it will be confirmed with a blood test during the first visit.
Babies who have no symptoms because they started treatment for UCD at birth and who also have a family history of UCD, may be able to take part in the trial.
Information about symptoms associated with having a Urea Cycle Disorder is available on FAQ: What are the symptoms associated with having a Urea Cycle Disorder?
Babies without UCD
Your baby can take part in the reLiver-1 trial if:
- They are in the 5th to 95th percentile of their age-related target bodyweight.
- They are healthy or they have an illness which is considered stable by the trial doctor.
For All Babies
Babies cannot participate if:
- They were born prematurely.
- They experienced significant weight loss after birth.
- They took part in another research trial within the last 30 days.
- They need to take medicines which might affect the trial results.
